Effects of Enzyme Replacement Therapy on Cardiac MRI Findings in Fabry Disease: A Systematic Review and Meta-Analysis

Stefano Figliozzi; Eleni Kollia; Alexandros Simistiras; Antonia Camporeale; Kamil Stankowski; Pier Giorgio Masci; George Mavraganis; Massimo Lombardi; Gianluigi Condorelli; Marco Francone; Maurizio Pieroni; Georgios Georgiopoulos

doi:10.1148/ryct.230154

Effects of Enzyme Replacement Therapy on Cardiac MRI Findings in Fabry Disease: A Systematic Review and Meta-Analysis

Stefano Figliozzi^*, Eleni Kollia, Alexandros Simistiras, Antonia Camporeale, Kamil Stankowski, Pier Giorgio Masci, George Mavraganis, Massimo Lombardi, Gianluigi Condorelli, Marco Francone, Maurizio Pieroni, Georgios Georgiopoulos

^*Corresponding author for this work

Research output: Contribution to journal › Article › peer-review

Abstract

Patients with Fabry disease undergoing enzyme replacement therapy showed stabilization of left ventricular mass and native T1 mapping, whereas the extent of late gadolinium enhancement slightly increased.

Purpose

To perform a systematic review and meta-analysis to assess the effect of enzyme replacement therapy on cardiac MRI parameters in patients with Fabry disease.

Materials and Methods

A systematic literature search was conducted from January 1, 2000, through January 1, 2024, in PubMed, ClinicalTrials.gov, Embase, and Cochrane Library databases. Study outcomes were changes in the following parameters: (a) left ventricular wall mass (LVM), measured in grams; (b) LVM indexed to body mass index, measured in grams per meters squared; (c) maximum left ventricular wall thickness (MLVWT), measured in millimeters; (d) late gadolinium enhancement (LGE) extent, measured in percentage of LVM; and (e) native T1 mapping, measured in milliseconds. A random-effects meta-analysis of the pooled mean differences between baseline and follow-up parameters was conducted. The study protocol was registered in PROSPERO (CRD42022336223).

Results

The final analysis included 11 studies of a total of 445 patients with Fabry disease (mean age ± SD, 41 years ± 11; 277 male, 168 female). Between baseline and follow-up cardiac MRI, the following did not change: T1 mapping (mean difference, 6 msec [95% CI: −2, 15]; two studies, 70 patients, I2 = 88%) and LVM indexed (mean difference, −1 g/m2 [95% CI: −6, 3]; four studies, 290 patients, I2 = 81%). The following measures minimally decreased: LVM (mean difference, −18 g [95% CI: −33, −3]; seven studies, 107 patients, I2 = 96%) and MLVWT (mean difference, −1 mm [95% CI: −2, −0.02]; six studies, 151 patients, I2 = 90%). LGE extent increased (mean difference, 1% [95% CI: 1, 1]; three studies, 114 patients, I2 = 85%).

Conclusion

In patients with Fabry disease, enzyme replacement therapy was associated with stabilization of LVM, MLVWT, and T1 mapping values, whereas LGE extent mildly increased.

Keywords: Fabry Disease, Enzyme Replacement Therapy (ERT), Cardiac MRI, Late Gadolinium Enhancement (LGE)

Original language	English
Article number	e230154
Journal	Radiology: Cardiothoracic Imaging
Volume	6
Issue number	3
Early online date	6 Jun 2024
DOIs	https://doi.org/10.1148/ryct.230154
Publication status	Published - Jun 2024

Keywords

Cardiac MRI
Enzyme Replacement Therapy (ERT)
Fabry Disease
Late Gadolinium Enhancement (LGE)

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10.1148/ryct.230154

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Figliozzi, S., Kollia, E., Simistiras, A., Camporeale, A., Stankowski, K., Masci, P. G., Mavraganis, G., Lombardi, M., Condorelli, G., Francone, M., Pieroni, M., & Georgiopoulos, G. (2024). Effects of Enzyme Replacement Therapy on Cardiac MRI Findings in Fabry Disease: A Systematic Review and Meta-Analysis. Radiology: Cardiothoracic Imaging, 6(3), Article e230154. https://doi.org/10.1148/ryct.230154

@article{97ed20dc43cc4d3a945e860ee1ce1460,

title = "Effects of Enzyme Replacement Therapy on Cardiac MRI Findings in Fabry Disease: A Systematic Review and Meta-Analysis",

abstract = "Patients with Fabry disease undergoing enzyme replacement therapy showed stabilization of left ventricular mass and native T1 mapping, whereas the extent of late gadolinium enhancement slightly increased.PurposeTo perform a systematic review and meta-analysis to assess the effect of enzyme replacement therapy on cardiac MRI parameters in patients with Fabry disease.Materials and MethodsA systematic literature search was conducted from January 1, 2000, through January 1, 2024, in PubMed, ClinicalTrials.gov, Embase, and Cochrane Library databases. Study outcomes were changes in the following parameters: (a) left ventricular wall mass (LVM), measured in grams; (b) LVM indexed to body mass index, measured in grams per meters squared; (c) maximum left ventricular wall thickness (MLVWT), measured in millimeters; (d) late gadolinium enhancement (LGE) extent, measured in percentage of LVM; and (e) native T1 mapping, measured in milliseconds. A random-effects meta-analysis of the pooled mean differences between baseline and follow-up parameters was conducted. The study protocol was registered in PROSPERO (CRD42022336223).ResultsThe final analysis included 11 studies of a total of 445 patients with Fabry disease (mean age ± SD, 41 years ± 11; 277 male, 168 female). Between baseline and follow-up cardiac MRI, the following did not change: T1 mapping (mean difference, 6 msec [95% CI: −2, 15]; two studies, 70 patients, I2 = 88%) and LVM indexed (mean difference, −1 g/m2 [95% CI: −6, 3]; four studies, 290 patients, I2 = 81%). The following measures minimally decreased: LVM (mean difference, −18 g [95% CI: −33, −3]; seven studies, 107 patients, I2 = 96%) and MLVWT (mean difference, −1 mm [95% CI: −2, −0.02]; six studies, 151 patients, I2 = 90%). LGE extent increased (mean difference, 1% [95% CI: 1, 1]; three studies, 114 patients, I2 = 85%).ConclusionIn patients with Fabry disease, enzyme replacement therapy was associated with stabilization of LVM, MLVWT, and T1 mapping values, whereas LGE extent mildly increased.Keywords: Fabry Disease, Enzyme Replacement Therapy (ERT), Cardiac MRI, Late Gadolinium Enhancement (LGE)",

keywords = "Cardiac MRI, Enzyme Replacement Therapy (ERT), Fabry Disease, Late Gadolinium Enhancement (LGE)",

author = "Stefano Figliozzi and Eleni Kollia and Alexandros Simistiras and Antonia Camporeale and Kamil Stankowski and Masci, {Pier Giorgio} and George Mavraganis and Massimo Lombardi and Gianluigi Condorelli and Marco Francone and Maurizio Pieroni and Georgios Georgiopoulos",

note = "Publisher Copyright: {\textcopyright} RSNA, 2024.",

year = "2024",

month = jun,

doi = "10.1148/ryct.230154",

language = "English",

volume = "6",

journal = "Radiology: Cardiothoracic Imaging",

issn = "2638-6135",

publisher = "Radiological Society of North America Inc.",

number = "3",

}

Figliozzi, S, Kollia, E, Simistiras, A, Camporeale, A, Stankowski, K, Masci, PG, Mavraganis, G, Lombardi, M, Condorelli, G, Francone, M, Pieroni, M & Georgiopoulos, G 2024, 'Effects of Enzyme Replacement Therapy on Cardiac MRI Findings in Fabry Disease: A Systematic Review and Meta-Analysis', Radiology: Cardiothoracic Imaging, vol. 6, no. 3, e230154. https://doi.org/10.1148/ryct.230154

TY - JOUR

T1 - Effects of Enzyme Replacement Therapy on Cardiac MRI Findings in Fabry Disease

T2 - A Systematic Review and Meta-Analysis

AU - Figliozzi, Stefano

AU - Kollia, Eleni

AU - Simistiras, Alexandros

AU - Camporeale, Antonia

AU - Stankowski, Kamil

AU - Masci, Pier Giorgio

AU - Mavraganis, George

AU - Lombardi, Massimo

AU - Condorelli, Gianluigi

AU - Francone, Marco

AU - Pieroni, Maurizio

AU - Georgiopoulos, Georgios

PY - 2024/6

Y1 - 2024/6

N2 - Patients with Fabry disease undergoing enzyme replacement therapy showed stabilization of left ventricular mass and native T1 mapping, whereas the extent of late gadolinium enhancement slightly increased.PurposeTo perform a systematic review and meta-analysis to assess the effect of enzyme replacement therapy on cardiac MRI parameters in patients with Fabry disease.Materials and MethodsA systematic literature search was conducted from January 1, 2000, through January 1, 2024, in PubMed, ClinicalTrials.gov, Embase, and Cochrane Library databases. Study outcomes were changes in the following parameters: (a) left ventricular wall mass (LVM), measured in grams; (b) LVM indexed to body mass index, measured in grams per meters squared; (c) maximum left ventricular wall thickness (MLVWT), measured in millimeters; (d) late gadolinium enhancement (LGE) extent, measured in percentage of LVM; and (e) native T1 mapping, measured in milliseconds. A random-effects meta-analysis of the pooled mean differences between baseline and follow-up parameters was conducted. The study protocol was registered in PROSPERO (CRD42022336223).ResultsThe final analysis included 11 studies of a total of 445 patients with Fabry disease (mean age ± SD, 41 years ± 11; 277 male, 168 female). Between baseline and follow-up cardiac MRI, the following did not change: T1 mapping (mean difference, 6 msec [95% CI: −2, 15]; two studies, 70 patients, I2 = 88%) and LVM indexed (mean difference, −1 g/m2 [95% CI: −6, 3]; four studies, 290 patients, I2 = 81%). The following measures minimally decreased: LVM (mean difference, −18 g [95% CI: −33, −3]; seven studies, 107 patients, I2 = 96%) and MLVWT (mean difference, −1 mm [95% CI: −2, −0.02]; six studies, 151 patients, I2 = 90%). LGE extent increased (mean difference, 1% [95% CI: 1, 1]; three studies, 114 patients, I2 = 85%).ConclusionIn patients with Fabry disease, enzyme replacement therapy was associated with stabilization of LVM, MLVWT, and T1 mapping values, whereas LGE extent mildly increased.Keywords: Fabry Disease, Enzyme Replacement Therapy (ERT), Cardiac MRI, Late Gadolinium Enhancement (LGE)

AB - Patients with Fabry disease undergoing enzyme replacement therapy showed stabilization of left ventricular mass and native T1 mapping, whereas the extent of late gadolinium enhancement slightly increased.PurposeTo perform a systematic review and meta-analysis to assess the effect of enzyme replacement therapy on cardiac MRI parameters in patients with Fabry disease.Materials and MethodsA systematic literature search was conducted from January 1, 2000, through January 1, 2024, in PubMed, ClinicalTrials.gov, Embase, and Cochrane Library databases. Study outcomes were changes in the following parameters: (a) left ventricular wall mass (LVM), measured in grams; (b) LVM indexed to body mass index, measured in grams per meters squared; (c) maximum left ventricular wall thickness (MLVWT), measured in millimeters; (d) late gadolinium enhancement (LGE) extent, measured in percentage of LVM; and (e) native T1 mapping, measured in milliseconds. A random-effects meta-analysis of the pooled mean differences between baseline and follow-up parameters was conducted. The study protocol was registered in PROSPERO (CRD42022336223).ResultsThe final analysis included 11 studies of a total of 445 patients with Fabry disease (mean age ± SD, 41 years ± 11; 277 male, 168 female). Between baseline and follow-up cardiac MRI, the following did not change: T1 mapping (mean difference, 6 msec [95% CI: −2, 15]; two studies, 70 patients, I2 = 88%) and LVM indexed (mean difference, −1 g/m2 [95% CI: −6, 3]; four studies, 290 patients, I2 = 81%). The following measures minimally decreased: LVM (mean difference, −18 g [95% CI: −33, −3]; seven studies, 107 patients, I2 = 96%) and MLVWT (mean difference, −1 mm [95% CI: −2, −0.02]; six studies, 151 patients, I2 = 90%). LGE extent increased (mean difference, 1% [95% CI: 1, 1]; three studies, 114 patients, I2 = 85%).ConclusionIn patients with Fabry disease, enzyme replacement therapy was associated with stabilization of LVM, MLVWT, and T1 mapping values, whereas LGE extent mildly increased.Keywords: Fabry Disease, Enzyme Replacement Therapy (ERT), Cardiac MRI, Late Gadolinium Enhancement (LGE)

KW - Cardiac MRI

KW - Enzyme Replacement Therapy (ERT)

KW - Fabry Disease

KW - Late Gadolinium Enhancement (LGE)

UR - http://www.scopus.com/inward/record.url?scp=85198719740&partnerID=8YFLogxK

U2 - 10.1148/ryct.230154

DO - 10.1148/ryct.230154

M3 - Article

AN - SCOPUS:85198719740

SN - 2638-6135

VL - 6

JO - Radiology: Cardiothoracic Imaging

JF - Radiology: Cardiothoracic Imaging

IS - 3

M1 - e230154

ER -

Effects of Enzyme Replacement Therapy on Cardiac MRI Findings in Fabry Disease: A Systematic Review and Meta-Analysis

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